Blog
RSS FeedPosted by Michael Wonder on 09 Apr 2024
ViiV Healthcare announces US FDA approval of Dovato (dolutegravir/lamivudine) for adolescents living with HIV
8 April 2024 - Dovato is now the first and only oral, two drug, single-tablet regimen available for people aged 12 ...
Posted by Michael Wonder on 02 Apr 2024
Solid Biosciences receives rare paediatric disease designation from the FDA for Duchenne muscular dystrophy gene therapy candidate SGT-003
1 April 2024 - Site initiations scheduled for April; patient dosing expected to begin in Q2 ,2024. ...
Posted by Michael Wonder on 30 Mar 2024
FDA Approves Medexus's supplemental biologics license application for Ixinity to treat haemophilia B in paediatric patients
26 March 2024 - Expanded indication includes patients under 12 years of age, based on Phase 3/4 data demonstrating safety and ...
Posted by Michael Wonder on 26 Mar 2024
Tonix Pharmaceuticals receives rare paediatric disease designation from the FDA for TNX-2900 for the treatment of Prader-Willi syndrome
25 March 2024 - TNX-2900 is a proprietary magnesium-potentiated formulation of intranasal oxytocin, a naturally occurring hormone that reduces appetite and ...
Posted by Michael Wonder on 25 Mar 2024
FDA grants Argenica rare paediatric disease designation for ARG-007 in the treatment of HIE
25 March 2024 - Argenica Therapeutics is pleased to announce the US FDA has granted its neuroprotective drug ARG-007 rare ...
Posted by Michael Wonder on 22 Mar 2024
Lisata Therapeutics announces US FDA rare paediatric disease designation granted to LSTA1 for the treatment of osteosarcoma
21 March 2024 - Lisata Therapeutics today announced that the US FDA has granted rare paediatric disease designation to LSTA1, the ...
Posted by Michael Wonder on 20 Mar 2024
A lifesaving therapy for children with a rare disease is now the world’s most expensive drug, raising questions about access
20 March 2024 - A new gene therapy for the fatal genetic disorder metachromatic leukodystrophy, or MLD, will carry a ...
Posted by Michael Wonder on 18 Mar 2024
FDA approves first gene therapy for children with metachromatic leukodystrophy
18 March 2024 - Today, the US FDA approved Lenmeldy (atidarsagene autotemcel), the first FDA approved gene therapy indicated for the ...
Posted by Michael Wonder on 14 Mar 2024
PepGen receives US FDA orphan drug and rare paediatric disease designations for PGN-EDO51 for the treatment of Duchenne muscular dystrophy
13 March 2024 - PepGen today announced that the US FDA granted both orphan drug and rare paediatric disease designations for ...
Posted by Michael Wonder on 11 Mar 2024
Praluent (alirocumab) injection receives FDA approval to treat children with genetic form of high cholesterol
11 March 2024 - Approval extends treatment of Praluent to children aged 8 and older with heterozygous familial hypercholesterolaemia. ...
Posted by Michael Wonder on 06 Mar 2024
FDA approves inotuzumab ozogamicin for paediatric patients with acute lymphoblastic leukaemia
6 March 2024 - Today, the FDA approved inotuzumab ozogamicin (Besponsa, Pfizer) for paediatric patients 1 year and older with ...
Posted by Michael Wonder on 23 Feb 2024
US FDA grants priority review to Harmony Biosciences' application for Wakix (pitolisant) in paediatric narcolepsy
21 February 2024 - Prescription Drug User Fee Act date is 21 June 2024. ...
Posted by Michael Wonder on 22 Feb 2024
Avidity Biosciences receives FDA rare paediatric disease designation for AOC 1044 for treatment of Duchenne muscular dystrophy in people with mutations amenable to exon 44 skipping
20 February 2024 - Avidity Biosciences today announced that the US FDA has granted rare paediatric disease designation to AOC 1044, ...
Posted by Michael Wonder on 15 Feb 2024
Dermavant submits supplemental new drug application to FDA for Vtama (tapinarof) 1% cream for the treatment of atopic dermatitis in adults and children 2 years of age and older
14 February 2024 - sNDA is supported by positive data from the Phase 3 ADORING 1 and ADORING 2 pivotal trials ...
Posted by Michael Wonder on 02 Feb 2024
Tyra Biosciences receives FDA rare paediatric disease designation for TYRA-300 for the treatment of achondroplasia
1 February 2024 - Tyra Biosciences today announced that the US FDA has granted rare paediatric disease designation to TYRA-300, an ...